Gene therapy company, AveXis, announced positive results from its ongoing SMA type 1 trial. This phase 1 trial is testing the safety and effectiveness of AVSX-101, an AAV-based therapy that aims to replace the defective SMN1 gene. Trial participants have shown improvements in their motor skills and there have been no major safety concerns so far. AveXis is due to meet with the FDA to discuss future developments for AVSX-101.
Biogen and Ionis Pharmaceuticals announced that infants with SMA type 1 who received nusinersen as part of the ongoing phase 3 trial called ENDEAR, have already experienced a statistically significant improvement in the achievement of motor milestones. The companies now intend to file for marketing approval with regulatory authorities in the coming months. Biogen is also working to open a global expanded access programme, which will allow all eligible infants with SMA type 1 to take nusinersen. We are seeking further information from the companies on implications for the UK and will share more details as soon as we have them.